7317 CAHtalyst Pediatric: Results From The Randomized, Double-Blind, Placebo-Controlled Period Of A Phase 3 Trial Of Crinecerfont, A Corticotropin-Releasing Factor Type 1 Receptor Antagonist, In Children And Adolescents With Classic Congenital Adrenal Hyperplasia

• Published in: Journal of the Endocrine Society 2024-10, Vol.8 (Supplement_1)
• Main Authors: Sarafoglou, K, Kim, M S, Lodish, M, Felner, E I, Martinerie, L, Nokoff, N J, Clemente, M, Fechner, P Y, Vogiatzi, M G, Speiser, P W, Sturgeon, J, Roberts, E, Jeha, G S, Chan, J L, Farber, R
• Format: Article
• Language: English
• Subjects: Abstract
• ISSN: 2472-1972; 2472-1972
• DOI: 10.1210/jendso/bvae163.127

Abstract Disclosure: K. Sarafoglou: Consulting Fee; Self; Neurocrine Biosciences, Inc., Crinetics Pharmaceuticals, Eton Pharmaceuticals, Spruce Biosciences. Research Investigator; Self; Neurocrine Biosciences, Inc., Adrenas Therapeutics, Spruce Biosciences. M.S. Kim: Advisory Board Member; Self; Spruce Biosciences. Research Investigator; Self; Neurocrine Biosciences, Inc., Diurnal, Spruce Biosciences. M. Lodish: None. E.I. Felner: None. L. Martinerie: None. N.J. Nokoff: Advisory Board Member; Self; World Athletics. Consulting Fee; Self; Ionis Pharmaceuticals Inc., Neurocrine Biosciences, Inc. M. Clemente: Consulting Fee; Self; AstraZeneca, Boehringer Ingelheim, Eli Lilly & Company, Novo Nordisk. Research Investigator; Self; Eli Lilly & Company, Novo Nordisk. P.Y. Fechner: Consulting Fee; Self; Neurocrine Biosciences, Inc., Eton Pharmaceuticals. Research Investigator; Self; Neurocrine Biosciences, Inc., Diurnal, Spruce Biosciences. M.G. Vogiatzi: Consulting Fee; Self; Adrenas Therapeutics, Eton Pharmaceuticals. Research Investigator; Self; Neurocrine Biosciences, Inc., Spruce Biosciences. P.W. Speiser: Advisory Board Member; Self; Adrenas Therapeutics, Chan Zuckerberg's Rare as One Initiative. Consulting Fee; Self; Roche Diagnostics. Research Investigator; Self; Neurocrine Biosciences, Inc.. Speaker; Self; Medscape, Wolters Kluter Publishing. J. Sturgeon: Employee; Self; Neurocrine Biosciences, Inc. E. Roberts: Employee; Self; Neurocrine Biosciences, Inc. G.S. Jeha: Employee; Self; Neurocrine Biosciences, Inc. J.L. Chan: Employee; Self; Neurocrine Biosciences, Inc. R. Farber: Employee; Self; Neurocrine Biosciences, Inc.. Introduction: Classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21OHD), a genetic disorder characterized by cortisol deficiency and excess adrenal androgens, typically requires management with supraphysiological glucocorticoid (GC) doses. Crinecerfont is a novel oral CRF1 antagonist that reduced elevated ACTH and adrenal androgens in patients with 21OHD in 14-day, open-label Phase 2 studies. This Phase 3 study (CAHtalyst Pediatric: NCT04806451) evaluated whether crinecerfont can reduce androstenedione (A4) levels and supraphysiological GC doses in pediatric patients with 21OHD. Methods: Children and adolescents (2-17 yrs) with classic 21OHD, stable GC regimens at doses >12 mg/m2/d in hydrocortisone equivalents (HCe), A4 >midpoint of reference range, and 17-hydroxyprogesterone (17-OHP) >2x upper limit of normal (